Top 6 Cystic fibrosis treatment startups in USA

Aug 29, 2026
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1
Oorja Bio
Funding: $30M
Oorja Bio advances in-licensed, novel approach to treat idiopathic pulmonary fibrosis. The biotech’s lead asset is ORJ-001, an activator of a receptor called β1 integrin that is key for maintaining the structure and stability of the lungs. A lack of β1 integrin can promote inflammation and lung damage. β1 integrin is found on important skin cells in the lung’s air sacs, called alveoli. These alveolar type 2 cells secrete molecules that help the lungs expand easily when breathing and protect the balloon-like organs from infection. ORJ-001 was formerly called NP-201 and was discovered by a South Korean biotech called NIBEC.
2
Sionna Therapeutics
Funding: $292M
Sionna’s batch of small molecules will be the first to fully restore function to the cystic fibrosis transmembrane conductance regulator (CFTR) protein
3
Mediar Therapeutics
Funding: $145.6M
Mediar is a pre-clinical stage biotechnology company developing therapeutics for the treatment of fibrosis.
4
Stablix Therapeutics
Funding: $63M
Stablix Therapeutics, a biotechnology company pioneering the Targeted Protein Stabilization technologies that is used to cure cystic fibrosis and cancer
5
Lung Therapeutics
Funding: $53.9M
Lung Therapeutics pursues innovative treatments for underserved, life-threatening lung conditions. Our therapies for Idiopathic Pulmonary Fibrosis and Loculated Pleural Effusion bring hope to patients whose only treatment options may be invasive surgery or costly and ineffective drugs. We aim to create better pharmaceutical treatment options for IPF, LPE, and other underserved lung conditions.
6
Carbon Biosciences
Funding: $38M
Carbon Biosciences is a biotech company that engages in the novel parvovirus gene therapy.
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Editor: Jason Kwon
Jason Kwon is a senior editor for MedicalStartups. He has previously covered the pharmaceutical and medical research industries for FDAnews and worked as a head of marketing for medical startup Sonic Therapeutics. Before that, he co-founded a startup consulting business for emerging entrepreneurial hubs in Asia. Jason graduated from St. Bonaventure University’s journalism school. In his free time, Jason enjoys yoga, watching movie trailers, traveling to places where he can't get cell service. You can contact Jason at jaskwon(at)medicalstartups(dot)com